Eplontersen approved in EU for the treatment of ATTRv-PN
Eplontersen has been approved in the EU for the treatment of hereditary transthyretin-mediated amyloidosis in adults with stage 1 or stage 2 polyneuropathy (ATTRv-PN).
Eplontersen has received approval from the EU for the treatment of hereditary transthyretin-mediated amyloidosis in adults with stage 1 or stage 2 polyneuropathy (hATTR-PN or ATTRv-PN). Eplontersen is currently the only approved self-administration drug for the treatment of ATTRv-PN. The approval was based on positive results from the phase 3 NEURO-TTRansform trial.
In NEURO-TTRansform, treatment with eplontersen for 66 weeks reduced serum TTR concentration and neuropathy impairment and improved quality of life in patients with ATTRv-PN, compared with placebo. The safety and tolerability profile of eplontersen in NEURO-TTRansform was favorable.
Eplontersen is an once-monthly RNA-targeted medicine that targets hepatic TTR production. The ongoing CARDIO-TTRansform phase 3 study evaluates the efficacy and safety of eplontersen in adults with ATTR cardiomyopathy.